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BIOPHYTIS Receives Orphan Drug Designation in the United States for Sarconeos in Duchenne Muscular Dystrophy (DMD)PARIS, May 15, 2018 (GLOBE NEWSWIRE) -- BIOPHYTIS (Euronext Growth Paris:ALBPS), a biotechnology company specializing in the development of drug candidates to treat age-related degenerative diseases, announces that the FDA (Food and Drug Administration) has granted orphan drug designation to its drug candidate Sarconeos in Duchenne Muscular Dystrophy (DMD). Stanislas Veillet, CEO of BIOPHYTIS, said: "The orphan drug designation that has just been granted by the FDA is a significant milestone for our team and the development of Sarconeos in Duchenne Myopathy, a particularly severe pediatric indication. We hope to obtain this designation in Europe as well in the coming weeks. We will then be able to conduct the MYODA clinical program. This program will consist of two studies: a pharmacokinetic phase 1/2a MYODA-PK study, which could begin in 2018 and an efficacy phase 2/3 MYODA-INT study, which could start in 2019.” The orphan drug designation by the FDA will grant Sarconeos with the benefits associated with the status, in particular, a 7 years protection after marketing authorization, possible co-financing of clinical trials with the FDA and the possibility of an accelerated registration procedure. BIOPHYTIS has also filed an application for an orphan drug designation for Sarconeos in Duchenne myopathy with the EMA (European Medicines Agency). The Agency’s response is expected in the coming weeks. Sarconeos’ proof-of-concept in the treatment of Duchenne Muscular Dystrophy has been presented at the World Muscle Society congress in 2017. It was demonstrated, in the reference animal model of Duchenne Muscular Dystrophy, that Sarconeos had significantly improved exercise tolerance and muscle strength, and reduced muscle fibrosis. Orphan drug designation is a first regulatory step to obtain authorizations to develop Sarconeos in Duchenne Muscular Dystrophy, in parallel with its development in Sarcopenia. It marks the implementation of a dual development strategy of BIOPHYTIS’ drug candidates, not only in chronic geriatric diseases, but also in genetic degenerative diseases. Sarconeos’ clinical development plan in Duchenne myopathy consists of two main clinical studies: a pharmacokinetic phase I/II MYODA-PK study to be initiated in 2018, and a phase II/III efficacy study, MYODA-INT, that could start in 2019. There are very few effective treatment options and Sarconeos is a new class of drug candidate that has the potential to significantly delay the progression of the disease. It could be used as a standalone treatment, or in combination with gene therapy when it will be available for children with Duchenne myopathy. About MYODA: About Duchenne muscular dystrophy: About SARCONEOS: About BIOPHYTIS BIOPHYTIS is listed on the Euronext Growth market of Euronext Paris For more information: http://www.biophytis.com Follow us on Twitter @biophytis BIOPHYTIS is eligible for the SMEs scheme Disclaimer This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in BIOPHYTIS in any country. Items in this press release may contain forward-looking statements involving risks and uncertainties. The Company’s actual results could differ substantially from those anticipated in these statements owing to various risk factors which are described in the Company’s prospectus. This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall prevail.
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